- Iovance Biotherapeutics said it doesn't expect to seek U.S. approval of the experimental cancer cell therapy lifileucel until 2022 after failing to convince regulators that its potency testing is adequate.
- The California biotech had once hoped to file its application with the Food and Drug Administration last year and made plans in 2019 to build a $75 million plant in Philadelphia to support production. After the FDA raised objections to Iovance's so-called potency assays — lab tests meant to measure the strength of its treatment — the company pushed back its target application date to 2021.
- Iovance said it's working to develop and validate new assays that will meet the FDA's requirements and now expects to submit the therapy for approval in the first half of next year. The company's shares dropped by more than 10% at the start of Wednesday trading and then fell further after the company separately disclosed its CEO will be resigning.
Lifileucel aims to treat cancer by using immune cells called tumor-infiltrating lymphocyte, or TILs. The therapy involves tinkering with a patient's own cells, which are extracted from a tumor, engineered, expanded and then infused back into the body. Though the approach is similar to the method used in multiple CAR-T treatments the FDA has already approved, there are some notable differences, possibly making Iovance's regulatory path more difficult.
As each patient's cells are different, measuring potency for the resulting therapy is difficult, Jefferies analyst Michael Yee wrote in a note to investors. The delays and uncertainty are frustrating for investors, but Yee expects the FDA ultimately "will appreciate the unmet need is high, the product is important, and this is something that can get worked out."
Data from clinical trials suggests lifileucel is effective in fighting melanoma and cervical cancer. Yee said he now expects approval for melanoma in mid-2023 and clearance for cervical cancer in the second half of 2023.
The issue is "likely resolvable," but risk is increasing for the stock, Stifel analyst Benjamin Burnett wrote in a note to investors. One fear is that additional clinical data might be required to go along with new potency assays.
The company's current CEO, Maria Fardis, won't be around to see the issue through, however. In a separate announcement Wednesday, Iovance disclosed that Fardis will resign to "pursue other opportunities." Frederick Vogt, the company's top lawyer, will serve as interim chief executive once Fardis departs.
There are signs that the FDA is taking a tougher line on gene therapy development in general. The agency last year asked Sarepta to run another laboratory test using a new potency assay for its experimental gene therapy for Duchenne muscular dystrophy. And Pfizer recently said it's working with the FDA to resolve questions about potency assays for its own experimental Duchenne treatment.
Note: This story has been updated to include mention of CEO Maria Fardis' resignation.